A Study of IMR-687 in Subjects With Beta Thalassemia

May 13, 2025 updated by: Cardurion Pharmaceuticals, Inc.

A Phase 2 Study to Evaluate the Safety and Tolerability of IMR-687 in Subjects With Beta Thalassemia

A Study to Evaluate the Safety and Tolerability of IMR-687 in Subjects with Beta Thalassemia

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Terminated

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

A phase 2, randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, PK, and PD of IMR-687 (phosphodiesterase (PDE) 9 inhibitor) administered once daily (qd) orally for 36 weeks in 2 populations of adult subjects with β-thalassemia: Population 1 (Transfusion Dependent Thalassemia (TDT) subjects) and Population 2 (Non-Transfusion Dependent Thalassemia (NTDT) subjects).

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
122

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Denmark
    • Hovedstaden
      • Herlev, Hovedstaden, Denmark, 2730
        • Herlev Hospital
  • France
      • Paris, France, 75015
        • Hôpital Necker-Enfants Malades
    • Haute-Garonn
      • Toulouse cedex 9, Haute-Garonn, France, 31059
        • Institut Universitaire du Cancer de Toulouse Oncopole
    • Rhone
      • Lyon Cedex 03, Rhone, France, 69437
        • Hôpital Edouard Herriot
  • Georgia
      • Tbilisi, Georgia, 0159
        • National Center of Surgery
      • Tbilisi, Georgia, 0186
        • Medinvest - Institute of Hematology and Transfusiology
    • Borjomi
      • Tbilisi, Borjomi, Georgia, 0112
        • M. Zodelava Hematology Centre
  • Greece
    • Attica
      • Athens, Attica, Greece, 11527
        • Aghia Sofia General Children's Hospital
      • Athens, Attica, Greece, 11527
        • Laiko General Hospital of Athens
    • Central Macedonia
      • Thessaloníki, Central Macedonia, Greece, 54642
        • Ippokrateio General Hospital of Thessaloniki
    • Peloponnese
      • Patra, Peloponnese, Greece, 26504
        • University General Hospital of Patras
  • Israel
      • Afula, Israel, 18101
        • Emek Medical Center
    • Haifa District
      • Haifa, Haifa District, Israel, 3109601
        • Rambam Health Care Campus
    • Jerusalem District
      • Jerusalem, Jerusalem District, Israel, 9112001
        • Hadassah University Hospital Ein Kerem
    • Northern District
      • Nahariya, Northern District, Israel, 2210001
        • The Galilee Medical Center
  • Italy
    • Turin
      • Orbassano, Turin, Italy, 10043
        • Azienda Ospedaliera Giuseppe Brotzu
      • Orbassano, Turin, Italy, 10043
        • Azienda Ospedaliera Universitaria - Università degli Studi della Campania Luigi Vanvitelli
  • Lebanon
      • Hazmiyeh, Lebanon, 213
        • Chronic Care Center
  • Malaysia
    • Johor
      • Johor Bahru, Johor, Malaysia, 80100
        • Hospital Sultanah Aminah Johor Bharu
    • Kedah
      • Alor Setar, Kedah, Malaysia, 05460
        • Hospital Sultanah Bahiyah
    • Penang
      • George Town, Penang, Malaysia, 10450
        • Hospital Pulau Pinang
    • Perak
      • Ipoh, Perak, Malaysia, 30450
        • Hospital Raja Permaisuri Bainun
    • Sabah
      • Kota Kinabalu, Sabah, Malaysia, 88586
        • Hospital Queen Elizabeth - Kota Kinabalu
    • Sarawak
      • Kuching, Sarawak, Malaysia, 93586
        • Hospital Umum Sarawak
  • Morocco
      • Rabat, Morocco, 10100
        • Hôpital d'Enfants Rabat
  • Netherlands
    • North Holland
      • Amsterdam, North Holland, Netherlands, 1105 AZ
        • Amsterdam Universitair Medische Centra - Academisch Medisch Centrum
  • Tunisia
      • Sousse, Tunisia, 4000
        • Centre Hôpital Universitaire Farhat Hached
      • Tunis, Tunisia, 1006
        • Centre National de Greffe de la Moelle Osseuse
      • Tunis, Tunisia, 1008
        • Hospital Aziza Othmana
  • Turkey
      • Ankara, Turkey, 06230
        • Hacettepe Universitesi
      • Izmir, Turkey, 35100
        • Ege Universitesi Tip Fakultesi
    • Icel
      • Mersin, Icel, Turkey, 33110
        • Akdeniz Universitesi
      • Mersin, Icel, Turkey, 33110
        • Mersin Üniversitesi Tıp Fakültesi
  • United Kingdom
    • England
      • London, England, United Kingdom, N19 5NF
        • Whittington Health NHS Trust
      • London, England, United Kingdom, NW1 2PG
        • University College London Hospitals Nhs Foundation Trust
      • Manchester, England, United Kingdom, M13 9WL
        • Manchester University NHS Foundation Trust

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years to 65 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Documented diagnosis of β-thalassemia or HbE/ β-thalassemia in their medical history. Concomitant alpha gene deletion, duplication, or triplication is allowed.
  2. Documentation of the dates of transfusion events and the number of all pRBC units per event within the 12 weeks prior to the Baseline (Day 1) visit. .
  3. Must be willing and able to complete all study assessments and procedures, and to communicate effectively with the investigator and site staff.
  4. TDT Subjects: subjects must be regularly transfused, defined as >3 to 10 pRBC units in the12 weeks prior to Baseline (Day 1) visit and no transfusion-free period for >35 days during that period.
  5. NTDT subjects: Subjects must be transfusion independent, defined as 0 to ≤3 units of pRBCs received during the 12-week period prior to the Baseline (Day 1) visit, must not be on a regular transfusion program, must be RBC transfusion-free for at least ≥ 4 weeks prior to randomization, and must not be scheduled to start a regular
  6. hematopoietic stem cell transplantation within 9 months.
  7. NTDT subjects: Subjects must have Hb ≤10.0 g/dL at Screening; the screening Hb sample must be collected 7 to 28 days prior to randomization. Hb values within 21 days post-transfusion will be excluded.
  8. ECOG performance score of 0 to 1
  9. Female subjects must not be pregnant, or breastfeeding and be highly unlikely to become pregnant. Male subjects must be unlikely to impregnate a partner.

Exclusion Criteria:

  1. Diagnosis of α-thalassemia (e.g., hemoglobin H [HbH]) or hemoglobin S (HbS)/ β thalassemia.
  2. Body mass index (BMI) <17.0 kg/m2 or a total body weight <45 kg; or BMI >35 kg/m2
  3. Subjects with known active hepatitis A, hepatitis B, or hepatitis C, with active or acute event of malaria, or who are known to be positive for human immunodeficiency virus (HIV).
  4. Stroke requiring medical intervention ≤24 weeks prior to randomization.
  5. Platelet count >1000 × 109/L.
  6. Participated in another clinical study of an investigational agent (or device) within 30 days or 5-half-lives of date of informed consent, whichever is longer, or is currently participating in another study.
  7. For Subjects on iron chelation therapy (ICT) at the time of ICF signing, initiation of ICT less than 24 weeks before the predicted randomization date.
  8. Prior exposure to sotatercept or luspatercept, IMR-687, or gene therapy within 6 months prior to randomization (Day 1).
  9. Subjects who have major organ damage

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Lower Dose IMR-687
Oral administration of once daily IMR-687
Drug: IMR-687
Oral administration of once daily IMR-687
Experimental: Higher dose IMR-687
Oral administration of once daily IMR-687
Drug: IMR-687
Oral administration of once daily IMR-687
Placebo Comparator: Placebo
Oral administration of once daily placebo
Drug: Placebo
Oral administration of once daily Placebo

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
IMR-687 Safety and Tolerability
Time Frame: Baseline to Week 40
Incidence and severity of Adverse Events Incidence and severity of Serious Adverse Events
Baseline to Week 40

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
TDT Patients: Reduction in Red Blood Cell (RBC) Transfusion Burden With ≥33% Hematological Improvement From Week 12 to Week 24
Time Frame: Baseline to Week 24
Proportion of patients with ≥33% hematological improvement (as measured by reduced transfusion burden) from Week 12 to Week 24 compared to the 12 weeks prior to Baseline (Day 1)
Baseline to Week 24
NTDT Patients: Proportion of Subjects With an Increase From Baseline of Hb at Week 12 to Week 24 in the Absence of Transfusions.
Time Frame: Baseline to Week 24
Proportion of subjects with an increase from baseline of ≥1.0 g/dL in mean Hb values at Week 12 to Week 24 in the absence of transfusions.
Baseline to Week 24
NTDT Patients: Proportion of Subjects With an Increase From Baseline of ≥3% in Mean HbF Values at Week 12 to Week 24 in Absence of Transfusions
Time Frame: Baseline to Week 24
Proportion of subjects with an increase from baseline of ≥3% in mean HbF values at Week 12 to Week 24 in absence of transfusions
Baseline to Week 24
TDT Patients: Reduction in Red Blood Cell (RBC) Transfusion Burden With ≥33% Hematological Improvement From Week 24 to Week 36
Time Frame: Baseline to Week 36
Proportion of patients with ≥33% hematological improvement from Week 24 to Week 36 compared to the 12 weeks prior to Baseline (Day 1)
Baseline to Week 36
TDT Patients: Reduction in Red Blood Cell (RBC) Transfusion Burden With ≥50 % Hematological Improvement From Week 12 to Week 24
Time Frame: Baseline to Week 24
Proportion of patients with ≥50% hematological improvement from Week 12 to Week 24 compared to the 12 weeks prior to Baseline (Day 1)
Baseline to Week 24
TDT Patients: Reduction in Red Blood Cell (RBC) Transfusion Burden With ≥50% Hematological Improvement From Week 24 to Week 36
Time Frame: Baseline to Week 36
Proportion of patients with ≥50% hematological improvement from Week 24 to Week 36 compared to the 12 weeks prior to Baseline (Day 1)
Baseline to Week 36
NTDT Patients: Proportion of Subjects With an Increase From Baseline of Hb at Week 24 to Week 36 in the Absence of Transfusions
Time Frame: Baseline to Week 36
Proportion of subjects with an increase from baseline of ≥1.0 g/dL in mean Hb values at Week 24 to Week 36 in the absence of transfusions.
Baseline to Week 36
NTDT: Proportion of Subjects With an Increase From Baseline of ≥3% in Mean HbF Values at Week 24 to Week 36 in Absence of Transfusions
Time Frame: Baseline to Week 36
Proportion of subjects with an increase from baseline of ≥3% in mean HbF values at Week 24 to Week 36 in absence of transfusions
Baseline to Week 36

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Cardurion Pharmaceuticals, Inc.

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
Imara, Inc.

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Steve Luperchio, Cardurion Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
October 16, 2020

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
March 11, 2022

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
May 4, 2022

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
April 15, 2020

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
May 27, 2020

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
June 2, 2020

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
May 15, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
May 13, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
May 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • IMR-BTL-201
  • 2019-002989-12 (EudraCT Number)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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